Lung gene therapy

Clinical and regulatory issues

Aniruddha C. Amrite, Narayan P S Cheruvu, Sneha Sundaram, Rajagopal N. Aravalli, Pi-Wan Cheng, Uday B. Kompella

Research output: Contribution to journalReview article

1 Citation (Scopus)

Abstract

Lung gene therapy is a promising therapeutic approach for several difficult to treat disorders such as cystic fibrosis, α1-antitrypsin deficiency, and cancers. Although several gene therapy protocols have proven success in preclinical studies, when moved to the clinical stages, they have met with limited success. Thus, there is a need to carefully assess the developmental approaches undertaken with gene therapy products intended for lung disorders. This review summarizes the advances made in lung gene therapy, discusses the limitations of the existing approaches including the lack of reliability of preclinical studies, immunogenecity and toxicity of the gene therapy vectors, and the poor efficiency of nonviral vectors, and invokes the role of ethics and the regulatory agencies in better developing the gene therapy products.

Original languageEnglish (US)
Pages (from-to)1-28
Number of pages28
JournalClinical Research and Regulatory Affairs
Volume21
Issue number1
DOIs
StatePublished - Jul 30 2004

Fingerprint

Genetic Therapy
Lung
Cystic Fibrosis
Ethics
Neoplasms

Keywords

  • FDA
  • Immunogenicity
  • Lung gene therapy
  • Nonviral vectors
  • Toxicity
  • Viral vectors

ASJC Scopus subject areas

  • Pharmacology
  • Pharmaceutical Science
  • Pharmacology (medical)

Cite this

Amrite, A. C., Cheruvu, N. P. S., Sundaram, S., Aravalli, R. N., Cheng, P-W., & Kompella, U. B. (2004). Lung gene therapy: Clinical and regulatory issues. Clinical Research and Regulatory Affairs, 21(1), 1-28. https://doi.org/10.1081/CRP-120039559

Lung gene therapy : Clinical and regulatory issues. / Amrite, Aniruddha C.; Cheruvu, Narayan P S; Sundaram, Sneha; Aravalli, Rajagopal N.; Cheng, Pi-Wan; Kompella, Uday B.

In: Clinical Research and Regulatory Affairs, Vol. 21, No. 1, 30.07.2004, p. 1-28.

Research output: Contribution to journalReview article

Amrite, AC, Cheruvu, NPS, Sundaram, S, Aravalli, RN, Cheng, P-W & Kompella, UB 2004, 'Lung gene therapy: Clinical and regulatory issues', Clinical Research and Regulatory Affairs, vol. 21, no. 1, pp. 1-28. https://doi.org/10.1081/CRP-120039559
Amrite, Aniruddha C. ; Cheruvu, Narayan P S ; Sundaram, Sneha ; Aravalli, Rajagopal N. ; Cheng, Pi-Wan ; Kompella, Uday B. / Lung gene therapy : Clinical and regulatory issues. In: Clinical Research and Regulatory Affairs. 2004 ; Vol. 21, No. 1. pp. 1-28.
@article{41d79891aabb4bf3b3b9f54586d79293,
title = "Lung gene therapy: Clinical and regulatory issues",
abstract = "Lung gene therapy is a promising therapeutic approach for several difficult to treat disorders such as cystic fibrosis, α1-antitrypsin deficiency, and cancers. Although several gene therapy protocols have proven success in preclinical studies, when moved to the clinical stages, they have met with limited success. Thus, there is a need to carefully assess the developmental approaches undertaken with gene therapy products intended for lung disorders. This review summarizes the advances made in lung gene therapy, discusses the limitations of the existing approaches including the lack of reliability of preclinical studies, immunogenecity and toxicity of the gene therapy vectors, and the poor efficiency of nonviral vectors, and invokes the role of ethics and the regulatory agencies in better developing the gene therapy products.",
keywords = "FDA, Immunogenicity, Lung gene therapy, Nonviral vectors, Toxicity, Viral vectors",
author = "Amrite, {Aniruddha C.} and Cheruvu, {Narayan P S} and Sneha Sundaram and Aravalli, {Rajagopal N.} and Pi-Wan Cheng and Kompella, {Uday B.}",
year = "2004",
month = "7",
day = "30",
doi = "10.1081/CRP-120039559",
language = "English (US)",
volume = "21",
pages = "1--28",
journal = "Clinical Research Practices and Drug Regulatory Affairs",
issn = "1060-1333",
publisher = "Informa Healthcare",
number = "1",

}

TY - JOUR

T1 - Lung gene therapy

T2 - Clinical and regulatory issues

AU - Amrite, Aniruddha C.

AU - Cheruvu, Narayan P S

AU - Sundaram, Sneha

AU - Aravalli, Rajagopal N.

AU - Cheng, Pi-Wan

AU - Kompella, Uday B.

PY - 2004/7/30

Y1 - 2004/7/30

N2 - Lung gene therapy is a promising therapeutic approach for several difficult to treat disorders such as cystic fibrosis, α1-antitrypsin deficiency, and cancers. Although several gene therapy protocols have proven success in preclinical studies, when moved to the clinical stages, they have met with limited success. Thus, there is a need to carefully assess the developmental approaches undertaken with gene therapy products intended for lung disorders. This review summarizes the advances made in lung gene therapy, discusses the limitations of the existing approaches including the lack of reliability of preclinical studies, immunogenecity and toxicity of the gene therapy vectors, and the poor efficiency of nonviral vectors, and invokes the role of ethics and the regulatory agencies in better developing the gene therapy products.

AB - Lung gene therapy is a promising therapeutic approach for several difficult to treat disorders such as cystic fibrosis, α1-antitrypsin deficiency, and cancers. Although several gene therapy protocols have proven success in preclinical studies, when moved to the clinical stages, they have met with limited success. Thus, there is a need to carefully assess the developmental approaches undertaken with gene therapy products intended for lung disorders. This review summarizes the advances made in lung gene therapy, discusses the limitations of the existing approaches including the lack of reliability of preclinical studies, immunogenecity and toxicity of the gene therapy vectors, and the poor efficiency of nonviral vectors, and invokes the role of ethics and the regulatory agencies in better developing the gene therapy products.

KW - FDA

KW - Immunogenicity

KW - Lung gene therapy

KW - Nonviral vectors

KW - Toxicity

KW - Viral vectors

UR - http://www.scopus.com/inward/record.url?scp=3242698947&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=3242698947&partnerID=8YFLogxK

U2 - 10.1081/CRP-120039559

DO - 10.1081/CRP-120039559

M3 - Review article

VL - 21

SP - 1

EP - 28

JO - Clinical Research Practices and Drug Regulatory Affairs

JF - Clinical Research Practices and Drug Regulatory Affairs

SN - 1060-1333

IS - 1

ER -